On December 2, 2019 Puma Biotechnology, Inc. (NASDAQ: PBYI), a biopharmaceutical company, reported that its licensing partner Specialised Therapeutics Asia (STA) has received marketing approval of NERLYNX (neratinib) in Singapore from the Health Sciences Authority (HSA), a statutory board under the Ministry of Health of the Singapore Government (Press release, Puma Biotechnology, DEC 2, 2019, View Source [SID1234551817]). NERLYNX is indicated for the extended adjuvant treatment of adult patients with early stage HER2-overexpressed/amplified breast cancer, to follow adjuvant trastuzumab-based therapy.

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STA Chief Executive Officer Carlo Montagner said, "The approval in Singapore is a key milestone for the company as NERLYNX is the first drug in our portfolio to be approved by the HSA for commercialization in Singapore. We look forward to extending the reach of this medicine, with regulatory dossiers also submitted in Malaysia, Brunei and the Philippines."

Alan H. Auerbach, Chief Executive Officer and President of Puma, added, "We have been extremely pleased with the strong commercial strategy implemented by Specialised Therapeutics in Australia and we look forward to their continued success throughout South East Asia. We are committed to expanding access to NERLYNX to breast cancer patients around the world."

About HER2-Positive Breast Cancer

Approximately 20 to 25 percent of breast cancer tumors over-express the HER2 protein. HER2-positive breast cancer is often more aggressive than other types of breast cancer, increasing the risk of disease progression and death. Although research has shown that trastuzumab can reduce the risk of early stage HER2-positive breast cancer returning after surgery, up to 25% of patients treated with trastuzumab experience recurrence.

On December 2, 2019 Cellectis (Euronext Growth: ALCLS – Nasdaq: CLLS), a clinical-stage biopharmaceutical company focused on developing immunotherapies based on gene-edited allogeneic CAR T-cells (UCART), reported that the first patient enrolled in the dose escalation Phase 1 clinical study for its UCART22 product candidate has been dosed at The University of Texas MD Anderson Cancer Center (Press release, Cellectis, DEC 2, 2019, View Source [SID1234551816]). This clinical study, BALLI-01, will evaluate the safety, expansion, persistence and clinical activity of UCART22, a product candidate composed of engineered T-cells expressing anti-CD22 chimeric antigen receptors, in patients with relapsed/refractory B-cell acute lymphoblastic leukemia (B-ALL).

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UCART22 targets CD22, which is a B-cell restricted surface antigen expressed in normal B-lineage cells, as well as in blast cells from B-ALL and other B-cell malignancies.

"By targeting the CD22 antigen, we aim at offering a therapeutic solution to patients living with B-ALL, including those patients that have relapsed or did not respond to CD19-directed therapy, such as CAR-T or bispecific antibody treatments. UCART22 is an allogeneic product candidate that can be dosed in all eligible patients even if they underwent prior autologous or allogeneic CAR-T therapies," said Dr. André Choulika, Chairman and CEO, Cellectis. "With the recent announcement of our UCARTCS1 first dosing, this milestone shows our momentum at Cellectis as we continue moving forward our portfolio of proprietary product candidates into clinical development. We have high hopes that UCART22 will address an unmet medical need for patients with B-ALL, with the potential to expand into other indications for B-cell malignancies."

The clinical trial is led by Dr. Nitin Jain, MD, Associate Professor, Department of Leukemia, Division of Cancer Medicine at The University of Texas MD Anderson Cancer Center (Houston, TX) in collaboration with Dr. Richard Larson, MD, Director of the Hematologic Malignancies Clinical Research Program at the University of Chicago Medicine (Chicago, IL) and with Dr. Gail Roboz, MD, Director, Clinical and Translational Leukemia Programs, Professor of Medicine, Weill Cornell Medical College The New York Presbyterian Hospital (New York, NY).

About UCART22

UCART22 is one of Cellectis’ wholly owned, allogeneic, off-the-shelf gene-edited T-cell product candidates, designed for the treatment of relapsed and refractory B-cell acute lymphoblastic leukemia (R/R B-ALL). Like CD19, CD22 is a cell surface antigen expressed from the pre-B-cell stage of development through mature B-cells. CD22 expression occurs in more than 90% of patients with B-ALL.

About B-cell Acute Lymphoblastic Leukemia (B-ALL)

B-cell acute lymphoblastic leukemia (B-ALL) is a hematologic disease characterized by the proliferation of immature lymphoid cells in the bone marrow, peripheral blood and other organs. The increase and accumulation of blast cells in the bone marrow results in suppression of the normal production of blood cell and blood plasma components, and can therefore cause anemia, thrombocytopenia, neutropenia and risk of infection. Acute lymphoblastic leukemia (ALL) can start either with early B-cells or T-cells at different stages of maturity, however, approximately 85% of ALL cases involve precursor B-cells (B-ALL).

On December 2, 2019 Oncopeptides AB (Nasdaq Stockholm: ONCO) reported that Joseph Horvat it has been appointed as President North America and CEO of Oncopetides Inc. Joseph will serve on the Management Team of Oncopeptides and will start today (Press release, Oncopeptides, DEC 2, 2019, View Source [SID1234551815]).

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The appointment of Joseph Horvat as President North America represents a significant milestone in the preparations for a potential US launch of melflufen in late 2020. With more than 23 years of pharmaceutical and biotech industry experience, Joseph brings in-depth knowledge spanning multiple therapeutic areas, brands, and geographies. With a thorough understanding of patients, customers, US payors and access issues, Joseph has achieved success across various marketing and sales management roles.

During the last nine years, he served in different positions at EMD Serono (a subsidiary of Merck KGaA, Darmstadt, Germany), most recently as Senior Vice President of the Oncology Business Unit with responsibility for driving the US oncology product portfolio. He previously played a critical role in the development of EMD Serono’s commercial build up in antizipation of an upcoming launch. As part of his responsibility, Joseph created a strong culture and attracted talent that incrementally added to the success of the franchise and set the foundation for EMD Serono’s launch of its first immuno-oncology product.

Prior to joining EMD Serono, Joseph spent over a decade of his early career at Bristol-Myers Squibb where he held various roles of increasing responsibility across several areas including oncology, cardiovascular and neuroscience.

Comment from CEO Jakob Lindberg
"We are pleased that Joseph will join us with his extensive experience of launching and marketing oncology products in the US. Oncopeptides is planning to submit a New Drug Application (NDA) for accelerated approval of melflufen at the end of the first quarter 2020. Preparations for a potential market approval in the US are accelerating and our US footprint is expanding rapidly. As President Oncopeptides North America, Joseph will be instrumental in building a first class US organization and executing a successful launch of melflufen. I also welcome Joseph to our Management Team and look forward to working together with him to bring melflufen to patients", said Jakob Lindberg, CEO of Oncopeptides.

For further information, please contact:

Jakob Lindberg, CEO of Oncopeptides
E-mail: [email protected]
Telephone: +46 8 615 20 40

Rein Piir, Head of Investor Relations at Oncopeptides
E-mail: [email protected]
Cell phone: +46 70 853 72 92

This information was submitted for publication at 08.00 CET December 2, 2019.

On December 2, 2019 Constellation Pharmaceuticals, Inc. (Nasdaq: CNST), a clinical-stage biopharmaceutical company using its expertise in epigenetics to discover and develop novel therapeutics, reported that it will host an analyst/investor event and webcast on December 9 at 12:30 PM EST in Salon 2 at the Rosen Centre Hotel in Orlando, Florida, in conjunction with the annual meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper) (Press release, Constellation Pharmaceuticals, DEC 2, 2019, View Source [SID1234551814]).

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The event will be webcast live and can be accessed on the Investor Relations section of Constellation’s website at View Source Analysts and investors may also access the event and participate in the live question-and-answer session by dialing (877) 473-2077 (domestic) or (661) 378-9662 (international) and referring to conference ID 7892044. A replay of the call will be available at (855) 859-2056, (404) 537-3406 or (800) 585-8367.

On December 2, 2019 Oncotelic Inc. ("Oncotelic"), a wholly owned subsidiary of Mateon Therapeutics Inc. (OTCQB:MATN, "Mateon") dedicated to the development of innovative treatments for cancer, reported the publication of two peer-reviewed expert editorials authored by Fatih Uckun MD PhD, the Chief Medical Officer for Mateon, and Vuong Trieu, PhD, the Chief Executive Officer and President for Mateon, at Oncotelic in the oncology journals Cancer Clinics Journal and Annals of Hematology and Oncology Research (Press release, Mateon Therapeutics, DEC 2, 2019, View Source [SID1234551813]). Also published was a peer-reviewed research article in the prestigious oncology journal Cancers.

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The editorial titled "Monotherapy of High-Grade Gliomas with a TGF-beta2 Targeting RNA Therapeutic" provides an overview regarding the development status, mechanism of action, and clinical potential of Mateon’s first-in-class RNA therapeutic OT101 for the treatment of the most aggressive brain tumors in adults.

Citation Reference: Uckun FM, Trieu VN. Monotherapy of High-Grade Gliomas with a TGF-beta2 Targeting RNA Therapeutic. Cancer Clin J. 2019; 1(1): 1007 (View Source)

The editorial titled "Convection-enhanced delivery of an anti-TGFbeta RNA therapeutic as a new therapeutic concept for children with diffuse intrinsic pontine glioma ("DIPG")" explains a new concept for the treatment of pediatric DIPG patients with an RNA therapeutic targeting TGFbeta 2 and the scientific rationale and proof of concept data behind it.

Citation Reference: Uckun FM, Trieu VN. Convection-enhanced delivery of an anti-TGFbeta RNA therapeutic as a new therapeutic concept for children with diffuse intrinsic pontine glioma. Annals of Hematology and Oncology Research 2019; 1(1):1003 (View Source)

"These editorials emphasize our commitment to advance our investigational drug OT101 which shows high clinical impact potential to address unmet needs in neuro-oncology.", stated Dr. Trieu.

The research article "Recurrent or Refractory High-Grade Gliomas Treated by Convection-Enhanced Delivery of a TGFβ2-Targeting RNA Therapeutic: A Post-Hoc Analysis with Long-Term Follow-Up" was published in Cancers as part of the Special Issue Personalized Medicine: Recent Progress in Cancer Therapy and is available online:

Abstract: View Source

HTML Version: View Source/htm

PDF Version: View Source/pdf

Special Issue: View Source

Citation Reference: Uckun, F.M.; Qazi, S.; Hwang, L.; Trieu, V.N. Recurrent or Refractory High-Grade Gliomas Treated by Convection-Enhanced Delivery of a TGFβ2-Targeting RNA Therapeutic: A Post-Hoc Analysis with Long-Term Follow-Up. Cancers 2019, 11, 1892 (View Source/htm)

Dr. Uckun explained: "Our post-hoc analysis of the NCT00431561 Phase II clinical study outcome data demonstrates that the anti-TGFβ2 RNA therapeutic OT101 when intratumorally-administered via convection-enhanced delivery (CED) exhibits promising single-agent activity against recurrent or refractory (R/R) high-grade gliomas (HGG). Most importantly, OT101 induced durable complete response (CR), partial response (PR) and stable disease (SD) in more than one third of the efficacy population. In the objective responders, OT101 displayed a previously not reported pattern of single agent activity, which was characterized by (i) slow but robust tumor size reductions; (ii) very often, an early onset transient increase of tumor edema and/or pseudo-progression which preceded the tumor size reductions; and (iii) late-onset objective responses that were achieved at a median of 287 days. To our knowledge, this is the first demonstration that the intratumoral delivery of an RNA therapeutic via extended CED in the absence of other therapeutic agents or radiation results in >3 year median PFS and >3.5 year OS in greater than one third of the efficacy population in a R/R HGG trial. It will be important to evaluate OT101 in difficult-to-treat IDH-wildtype, MGMT-unmethylated R/R HGG patients who have failed a temozolomide-based first line adjuvant therapy, as they have a dismal prognosis and are in urgent need for new and effective salvage therapies. Intratumorally administered OT101 exhibited a promising safety profile, but some CED procedure and device-related potential complications were also identified. Therefore, risk mitigation strategies, especially preventive methods aimed at reducing the risk of infections, will be employed in future trials to further improve the potential patient benefit of intratumoral OT101 therapy"

OT101, a first-in-class RNA therapeutic designed to abrogate the immunosuppressive actions of TGF-beta 2, is Oncotelic’s lead anti-brain tumor drug candidate. In a completed Phase 2 clinical study, OT-101 exhibited clinically meaningful single-agent activity and induced durable complete and partial responses in recurrent and refractory adult high-grade glioma patients, including adults with GBM. DIPG, an orphan disease with a low survival rate and no established or effective standard of care. Despite numerous clinical trials of chemotherapeutic agents, immuno-oncology drugs and specific targeted therapies, no significant progress has been made in the treatment of DIPG and the prognosis remains dismal, with a mean OS of 9–12 months from the time of diagnosis, a median survival time of approximately 10 months, and a two-year OS rate of less than 10 percent. Five-year survival is less than 3 percent, and many long-term survivors have evidence of moderate or severe cognitive impairment, likely as a consequence of radiation therapy. Chemotherapy does not have an established role in the management of patients with DIPG. Furthermore, there is no standard treatment for progressive DIPG after the failure of radiation therapy and no salvage regimen has been shown to extend survival. Therefore, there is an urgent need for therapeutic innovations for treatment of DIPG, as reflected by multiple treatment modalities being evaluated in early neuro-oncology clinical trials. Further development of OT-101 may offer renewed hope for salvage therapy not only for adult high-grade glioma patients but also for pediatric DIPG patients. Last month, US Food and Drug Administration (FDA) granted Rare Pediatric Disease Designation for OT101/Trabedersen for the treatment of diffuse intrinsic pontine glioma (DIPG) as a drug for a "rare pediatric disease," as defined in section 529(a)(3) of the Federal Food, Drug, and Cosmetic Act.